Pfizer buys into gene therapy
pharmafile | December 9, 2014 | News story | Research and Development, Sales and Marketing | London, Pfizer, gene therapy, haemophilia, spark, therapeutics
Pfizer has announced that it is establishing a gene therapy platform as it looks to expand its rare diseases operations.
As part of these efforts the company has signed a haemophilia research agreement with US biotech firm Spark Therapeutics, and has appointed gene therapy expert Michael Linden to head its new activities.
“The fundamental understanding of the biology of hereditary rare diseases, coupled with advances in the technology to harness disarmed viruses as gene delivery vehicles, provide a ripe opportunity to investigate the next wave of potential life-changing therapies for patients,” says Mikael Dolsten, Pfizer’s president of worldwide research and development.
“By establishing our gene therapy capabilities, we hope to gain a deeper understanding of the mechanisms that could potentially bring true disease modification for those suffering from devastating haematologic and neuromuscular diseases.”
Pfizer will work to co-develop Spark’s SPK-FIX, an Adeno-Associated Virus (AAV) vector programme for the potential treatment of haemophilia B – an area Pfizer is already well-established in. Spark will receive $20 million upfront with the potential for milestone payments worth up to $260 million.
Adeno-Associated Viruses are small viruses containing single stranded DNA that can insert genetic material into a specific chromosome with near 100% certainty.
SPK-FIX is expected to enter Phase I/II clinical trials in the first half of 2015. Under the terms of the agreement, Spark will have responsibility for clinical development through these studies while Pfizer will assume responsibility for pivotal trials, any regulatory approvals, and potential global commercialisation.
Geno Germano, Pfizer’s group president of global innovative pharma business, comments: “We believe the SPK-FIX program could add to our existing portfolio of hemophilia products and could pioneer a potential new treatment technology for patients with bleeding disorders.”
Pfizer has also brought in AAV expert Michael Linden, a professor at King’s College London and director of the University College London Gene Therapy Consortium. Linden will be with the company for a two-year secondment to lead its gene therapy research.
This is not Pfizer’s first foray into gene therapy, though – in August the company teamed up with Google-backed genetic testing firm 23andMe to find genetic causes for inflammatory bowel disorder. 23andMe has since been banned by the FDA and generated controversy in the UK, however.
George Underwood
Related Content
Cambridge’s Grassroots Life Sciences Showcase Branches Out to Oxford & London
Cambridge Wide Open Week (CWOW) returns from 11-19 June 2026, sharing the spirit and buzz …
Rare diseases – spinal muscular atrophy
Spinal muscular atrophy (SMA) is a rare disease characterised by voluntary muscle weakness that can …
Five Facts about spinal muscular atrophy
1 Spinal muscular atrophy (SMA) is a genetic disease involving the loss of motor neurons …
